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CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo
Huntington disease (HD) is a fatal dominantly inherited neurodegenerative disorder caused by CAG repeat expansion (>36 repeats) within the first exon of the huntingtin gene. Although mutant huntingtin (mHTT) is ubiquitously expressed, the brain shows robust and early degeneration. Current RNA int...
| Autores principales: | , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
American Society of Gene & Cell Therapy
2017
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5363210/ https://www.ncbi.nlm.nih.gov/pubmed/28129107 http://dx.doi.org/10.1016/j.ymthe.2016.11.010 |